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Therapeutic Oligonucleotides Targeting Liver Disease: TTR Amyloidosis

The liver has become an increasingly interesting target for oligonucleotide therapy. Mutations of the gene encoding transthyretin (TTR), expressed in vast amounts by the liver, result in a complex degenerative disease, termed familial amyloid polyneuropathy (FAP). Misfolded variants of TTR are linke...

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Detalles Bibliográficos
Autores principales: Niemietz, Christoph, Chandhok, Gursimran, Schmidt, Hartmut
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2015
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6332041/
https://www.ncbi.nlm.nih.gov/pubmed/26437390
http://dx.doi.org/10.3390/molecules201017944